A Prescription Drug User Fee Act target date of September 22, 2026 has been set for the application. The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA ...

Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been ...

Findings showed zilganersen 50mg demonstrated a significant improvement from baseline in the 10MWT compared with placebo at week 61. Topline data were announced from a pivotal study evaluating ...

CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (IONS) (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug ...

CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal study of zilganersen in children and adults living with ...

Alexander disease (AxD) is a rare neurodegenerative disorder arising from mutations in the gene encoding glial fibrillary acidic protein (GFAP). GFAP plays a critical role in maintaining astrocyte ...

Ionis Pharmaceuticals (NASDAQ:IONS) stock climbed 5% following the announcement of encouraging topline results from a pivotal study of zilganersen in patients with Alexander disease, a rare and often ...

– First and only investigational medicine for this rare, often fatal neurological condition – – On track to submit new drug application (NDA) in Q1 2026 – CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis ...